QUÉBEC CITY, Nov. 6, 2014 /CNW
Telbec/ - Aeterna Zentaris Inc. (NASDAQ: AEZS, TSX: AEZ) (the
"Company") today announced that the Company has received a Complete
Response Letter ("CRL") from the U.S. Food and Drug Administration
("FDA") for its New Drug Application ("NDA") for
MacrilenTM (macimorelin), a novel orally-active ghrelin
agonist, for use in evaluating adult growth hormone deficiency
("AGHD"). Based on its review, the FDA has determined that the NDA
cannot be approved in its present form.
The CRL mentions that the planned analysis of the Company's
pivotal trial did not meet its stated primary efficacy objective as
agreed to in the Special Protocol Assessment agreement letter
between the Company and the FDA. The CRL further mentioned issues
related to the lack of complete and verifiable source data for
determining whether patients were accurately diagnosed with AGHD.
The FDA concluded that, "in light of the failed primary
analysis and data deficiencies noted, the clinical trial does not
by itself support the indication." To address the deficiencies
identified above, the CRL states that the Company will need to
demonstrate the efficacy of macimorelin as a diagnostic test for
growth hormone deficiency in a new, confirmatory clinical
study.
The CRL also outlined that a serious event of electrocardiogram
QT interval prolongation occurred for which attribution to drug
could not be excluded. Therefore a dedicated thorough QT study to
evaluate the effect of macimorelin on the QT interval would be
necessary.
David Dodd, Chairman and CEO at
Aeterna Zentaris said, "Following the FDA's decision, we are
currently reviewing the outstanding issues stated in the CRL in
order to evaluate our options and future plans for
MacrilenTM."
Conference call
Management will be hosting a conference call for the investment
community beginning at 9:00 am (Eastern
Time), on Friday, November 7,
2014, to discuss MacrilenTM's NDA.
Individuals interested in participating in the live conference call
by telephone may dial, in Canada,
514-807-9895 or 647-427-7450, outside Canada, 888-231-8191. They may also listen
through the Internet at www.aezsinc.com in the "Newsroom" section.
A replay will be available on the Company's website for 30 days
following the live event.
About Adult Growth Hormone Deficiency (AGHD)
AGHD affects approximately 75,000 adults across the U.S.,
Canada and Europe. Growth hormone not only plays an
important role in growth from childhood to adulthood, but also
helps promote a hormonally-balanced health status. AGHD mostly
results from damage to the pituitary gland. It is usually
characterized by a reduction in bone mineral density, lean body
mass, exercise capacity, and overall quality of life.
About MacrilenTM (macimorelin)
Macimorelin, a ghrelin agonist, is a novel orally-active small
molecule that stimulates the secretion of growth hormone.
Macimorelin, under the trade name MacrilenTM, has been
granted orphan drug designation by the FDA for AGHD. Aeterna
Zentaris owns the worldwide rights to this novel patented
compound.
About Aeterna Zentaris
Aeterna Zentaris is a specialty biopharmaceutical company
engaged in developing and commercializing novel treatments in
oncology and endocrinology. For more information, visit
www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made
pursuant to the safe harbor provisions of the US Securities
Litigation Reform Act of 1995. Forward-looking statements involve
known and unknown risks and uncertainties that could cause the
Company's actual results to differ materially from those in the
forward-looking statements. Such risks and uncertainties include,
among others, the availability of funds and resources to pursue
R&D projects, the successful and timely completion of clinical
studies, the risk that safety and efficacy data from any of our
Phase 3 trials may not coincide with the data analyses from
previously reported Phase 1 and/or Phase 2 clinical trials, the
ability of the Company to efficiently commercialize one or more of
its products or product candidates, the ability of the Company to
take advantage of business opportunities in the pharmaceutical
industry, uncertainties related to the regulatory process, the
specific outcome of our recently announced global resources
optimization program and the financial impact on the Company
resulting therefrom, and general changes in economic conditions.
Investors should consult the Company's quarterly and annual filings
with the Canadian and US securities commissions for additional
information on risks and uncertainties relating to forward-looking
statements. Investors are cautioned not to rely on these
forward-looking statements. The Company does not undertake to
update these forward-looking statements. We disclaim any obligation
to update any such factors or to publicly announce the result of
any revisions to any of the forward-looking statements contained
herein to reflect future results, events or developments, unless
required to do so by a governmental authority or by applicable
law.
SOURCE Aeterna Zentaris Inc.